Long-term Follow-up of Participants Dosed With an Investigational Gene Editing Therapy for Cardiovascular Disease
This study, called LTF-001, is a long-term check-up for people who previously received an experimental gene-editing treatment. This treatment was designed to help with serious heart conditions like atherosclerotic cardiovascular disease, which causes hardening of the arteries, and types of high cholesterol, such as heterozygous familial hypercholesterolemia. Researchers want to understand the long-term effects of this new therapy. This means they will follow participants for 15 years after they first received the gene-editing treatment. The aim is to see if the treatment continues to be safe and effective over time, and to learn as much as possible about how it impacts people's health in the long run. This information is crucial for developing new and better ways to treat these heart conditions.
At a glance
What is this study about?
This study is about carefully watching people over a long time (up to 15 years) who have already had a new kind of treatment called 'gene editing therapy'. This treatment is still in its experimental stages, meaning it's being tested to see how well it works and if it's safe.
The experimental treatment was given to help with certain heart and blood vessel problems, specifically conditions where people have very high cholesterol because of their genes, or 'atherosclerotic cardiovascular disease', which is a build-up of fats in the arteries. By changing specific genes, doctors hope to help the body work better to reduce harmful cholesterol and improve heart health.
Following people for such a long time helps doctors understand if the gene therapy has lasting benefits and if there are any side effects that might appear much later. This long-term information is really important to ensure that new treatments are not only good for a short time but also safe and helpful in the years to come.
Key takeaways
- This study tracks people who received an experimental gene therapy.
- It aims to understand the long-term safety and effects of the therapy.
- Participation involves monitoring your health for up to 15 years.
- You must have already received the gene therapy in a previous trial to qualify.
- The findings will help improve future treatments for heart conditions.
Who may be eligible?
To be part of this study, you must be at least 18 years old. The main rule is that you must have already taken part in a previous study where you received at least one dose of the experimental gene-editing treatment being developed by the same company.
Also, you need to agree to join this specific long-term follow-up study. Before you can join, a doctor or nurse will explain everything about the study, and you'll need to give your 'informed consent', which means you understand what's involved and agree to take part.
- Are you 18 years old or older?
- Have you previously received the experimental gene-editing therapy from the study sponsor?
- Are you willing and able to give your informed consent to participate?
- Are you able to attend follow-up visits and assessments as required?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part, you will be followed for a total of 15 years. This doesn't mean you'll be in a clinic every day! The study is about checking in on your health over this long period. You will likely have regular check-ups, which could include doctor visits, blood tests, and other assessments to see how you are doing and to monitor the effects of the gene therapy. The schedule for these visits will be explained to you in detail. There are no new medications given in this particular study; it's purely about observing people who have already had the treatment.
Potential risks and benefits
Locations (3)
- Clinical Study CenterAuckland, New Zealand
- Clinical Study CenterChristchurch, New Zealand
- Clinical Study CenterLondon, United Kingdom
Common questions
What is gene-editing therapy?
It's a new type of treatment that aims to correct or change specific genes in your body to help treat certain diseases.
Why is this study 15 years long?
Because gene therapy is new, doctors want to make sure they understand its long-term effects on your health and safety.
Do I get new treatment in this study?
No, this study is only for observation. You would have received the experimental gene therapy in a previous study.
What are the common heart conditions this therapy tried to help?
It targeted conditions like high cholesterol due to genes (familial hypercholesterolemia) and hardening of the arteries (atherosclerotic cardiovascular disease).
Can I leave the study if I change my mind?
Yes, you can withdraw from the study at any time without it affecting your regular medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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